261st ENMC International Workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofddorp, The Netherlands-Reference-Cited by-同舟云学术

261st ENMC International Workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofddorp, The Netherlands

Published:2023-11 Issue:11 Volume:33 Page:884-896
ISSN:0960-8966
Container-title:Neuromuscular Disorders
language:en
Short-container-title:Neuromuscular Disorders

Author:

Servais Laurent^ORCID,Horton Rebecca,Saade Dimah,Bonnemann Carsten,Muntoni Francesco,Adjali Dr Oumeya,Beggs Dr Alan,Bharucha Dr Diana,Bönnemann Dr Carsten,Braun Dr Serge,Byrne Dr Barry,Corti Dr Manuela,Buj-Bello Dr Ana,Chamberlain Dr Jeff,Ferreiro Dr Ana,Flanigan Dr Kevin,Germanenko Mrs Olga,Goemans Dr Nathalie,Grant Dr Daniel,Hopkins Dr Sam,Horton Dr Rebecca,Kollb-Sielecka Dr Marta,Le Guiner Dr Caroline,Levy Dr. Dan,Lek Dr Angela,Miller Dr Weston,Morris Dr Carl,Dreghici Dr Roxana,Muntoni Dr Francesco,Saade Dr Dimah,Servais Dr Laurent,Singh Dr Teji,Vroom Drs Elisabeth,Wagner Dr Kathryn,Van Ieperen Mr Frank

Publisher

Elsevier BV

Subject

Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health

Reference32 articles.

1. A systematic review of adeno-associated virus gene therapies in neurology: the need for consistent safety monitoring of a promising treatment;Horton;J Neurol Neurosurg Psychiatry,2022

2. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response;Manno;Nat Med,2006

3. Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates;Nathwani;Blood,2007

4. Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an adeno-associated virus vector expressing human SMN;Hinderer;Hum Gene Ther,2018

5. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B;Nathwani;N Engl J Med,2011

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